Taysha Announcement Key Takeaways

On June 18th, Taysha shared interim clinical data from the REVEAL Adolescent & Adult and the REVEAL Pediatric study which are both evaluating TSHA-102, an investigational gene therapy for Rett syndrome.

You can read their press release here.

Here are our key takeaways:

Adults and adolescents trial –

There have been no serious adverse events (SAEs) in patients 1 and 2 as of 52- and 36-weeks post-dosing.

Based on long-term data, taken at 52 weeks post dosing (patient 1) and 25 weeks post-dosing (patient 2), both patients are showing sustained and new clinical improvements.

In addition, clinical observations reported by the Principal Investigator showed sustained and new improvements in motor skills, communication/socialisation, autonomic function and seizures.

Children’s trial –

There have been no serious adverse events (SAEs) in patients 1 and 2 as of 22 and 11 weeks post-dosing.

One participant experienced two SAEs that were not deemed treatment-related (both were related to underlying disease, and one was also attributed to immunosuppression) and have bees resolved.

Based on long-term data, taken at 12 weeks post dosing (patient 1) and 8 weeks post-dosing (patient 2), both patients are showing sustained and new clinical improvements.

Clinical observations reported by the Principal Investigator showed improvements in motor skills, communication/socialisation, autonomic function and seizures.

Next milestones to look for:

Initial data from cohort 2 (high dose) of the adult and adolescent trial in second half of 2024.

FAQs

What is the U.K. MHRA Innovative Licensing and Access Pathway (ILAP)?

The ILAP designation aims to make it easier and quicker for UK patients to access new treatments by speeding the time it takes to get new treatments to market by enhancing interactions with the UK regulator and other relevant bodies involved in the process.

Has the UK children’s trial started yet?

Although Taysha has received clearance from the UK regulator, the MHRA, the first UK trial site has not been activated and is not yet recruiting. More information about the UK children’s trial including inclusion and exclusion criteria can be found here.

In the first instance, numbers needed for UK Rett syndrome gene therapy trials will be very small and clinicians are likely to recruit from their patient list. As trials expand, it is likely that Reverse Rett will support trial recruitment through the Rett Registry UK. If the person you care for (of any age) is not registered, please register today. www.rettregistry.uk  and email to express interest in gene therapy trials [email protected]  

Will families who live outside the UK be eligible to participate?

Ultimately the lead doctor at any given trial site will decide the eligibility of an individual patient for the clinical trial. Though this varies site to site, participants are typically selected for enrolment using several considerations, including how close they live to the clinical trial site.

Rett families and/or their treating physician may contact a participating site directly to ask about potential trial participation.

At Reverse Rett, we encourage patients outside the UK who wish to take part in clinical trials and/or access emerging treatments to register on the Rett Registry UK so that we are able to gather and share information about where there are concentrations of patients with companies and other relevant bodies.